Eligibility, male only, with Duchenne Muscular Dystrophy. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants With Treatment Emergent Adverse Events (TEAEs)Primary· Baseline up to Week 89
TEAE: any untoward medical occurrence or undesirable event(s) that begins or worsens following administration of the study drug, whether or not considered related to the treatment by the Investigator. Severity of an adverse event (AE) was classified as: mild (does not interfere with usual function), moderate (interferes with usual function; may require medical intervention), severe (interferes significantly with usual function; likely require medical intervention), life-threatening, and fatal. Drug-related AEs: AEs with a possible or probable relationship to study drug. Serious AEs: death, a l
At least 1 TEAE
Group
Value
95% CI
Ataluren
36
Mild TEAE
Group
Value
95% CI
Ataluren
9
Moderate TEAE
Group
Value
95% CI
Ataluren
16
Severe TEAE
Group
Value
95% CI
Ataluren
10
Life-Threatening TEAE
Group
Value
95% CI
Ataluren
1
Fatal TEAE
Group
Value
95% CI
Ataluren
0
Related TEAE
Group
Value
95% CI
Ataluren
28
Serious TEAE
Group
Value
95% CI
Ataluren
6
Change From Baseline in 6-Minute Walk Distance (6MWD) as Measured by the 6-minute Walk Test (6MWT)Secondary· Baseline, Week 48 and Week 60
The 6MWD was assessed in participants who were ambulatory using standardized procedures. Participants were not permitted to use assistive devices during the 6MWD test. Only the results of the participant's best valid test at each visit were included in the analysis. The mean change from baseline in the distance the participant walked is reported.
Baseline
Group
Value
95% CI
Ataluren
367.8
± 107.3
Change from Baseline at Week 48
Group
Value
95% CI
Ataluren
-80.4
± 59.5
Change from Baseline at Week 60
Group
Value
95% CI
Ataluren
-101.5
± 64.4
Change From Baseline in Proximal Muscle Function as Assessed by Speed During Timed Function TestsSecondary· Baseline, Week 48 and Week 60
Timed function tests included time to stand from supine position (rise to standing), time to run/walk 10 meters (m), and time to ascend/descend 4 stairs. Timed function tests were assessed in ambulatory participants. A decrease from baseline reflects faster completion of the functional task and, thus, better muscle function. If the time taken to perform a test exceeded 30 seconds or if a participant could not perform the test due to disease progression (PD), a value of 30 seconds was used. Test results were set to missing in the analysis for participants who could not perform the tests for rea
Rise to Standing, Baseline
Group
Value
95% CI
Ataluren
13.57
± 11.111
Rise to Standing, Change from Baseline at Week 48
Group
Value
95% CI
Ataluren
6.49
± 8.059
Rise to Standing, Change from Baseline at Week 60
Group
Value
95% CI
Ataluren
5.77
± 7.790
Walk/Run 10 m, Baseline
Group
Value
95% CI
Ataluren
7.62
± 3.641
Walk/Run 10 m, Change from Baseline at Week 48
Group
Value
95% CI
Ataluren
3.15
± 5.591
Walk/Run 10 m, Change from Baseline at Week 60
Group
Value
95% CI
Ataluren
3.20
± 5.930
Ascend 4 Stairs, Baseline
Group
Value
95% CI
Ataluren
7.16
± 7.481
Ascend 4 Stairs, Change from Baseline at Week 48
Group
Value
95% CI
Ataluren
4.92
± 6.856
Change From Baseline in Standing From Supine Position as Assessed by Method Scores During Timed Function TestsSecondary· Baseline, Week 48 and Week 60
Timed test evaluations included scoring of method that ambulatory participants used to complete test. Scale for method used for standing from supine position: 1) Unable to stand from supine, even with use of a chair. 2) Assisted Gowers, requires furniture to rise from supine to full upright posture. 3) Full Gowers, rolls over, stands with both hands "climbing up" legs to above knees to achieve full upright posture. 4) Half Gowers, rolls over, stands up with 1 hand support on lower legs. 5) Rolls to side and/or stands with 1 or both hands on floor to start to rise but does not touch legs. 6) St
Baseline
Group
Value
95% CI
Ataluren
3.25
± 1.260
Change from Baseline at Week 48
Group
Value
95% CI
Ataluren
-0.58
± 0.929
Change from Baseline at Week 60
Group
Value
95% CI
Ataluren
-0.62
± 0.921
Change From Baseline in Run/Walk 10-Meters as Assessed by Method Scores During Timed Function TestsSecondary· Baseline, Week 48 and Week 60
Timed test evaluations included scoring of method that ambulatory participants used to complete test. Scale for method used to run/walk 10-meters: 1) Unable to walk independently. 2) Unable to walk independently but can walk with knee-ankle-foot orthoses (KAFOs) or with support from a person 3) Highly adapted, wide-based lordotic gait, cannot increase walking speed. 4) Moderately adapted gait, can pick up speed but cannot run. 5) Able to pick up speed but runs with a double stance phase (that is, cannot achieve both feet off the ground). 6) Runs and gets both feet off the ground (with no doubl
Baseline
Group
Value
95% CI
Ataluren
4.42
± 0.974
Change from Baseline at Week 48
Group
Value
95% CI
Ataluren
-0.25
± 1.073
Change from Baseline at Week 60
Group
Value
95% CI
Ataluren
-0.57
± 1.207
Change From Baseline in Ascending 4 Stairs as Assessed by Method Scores During Timed Function TestsSecondary· Baseline, Week 48 and Week 60
Timed test evaluations included scoring of method that ambulatory participants used to complete test. Scale for method used to ascend 4 stairs: 1) Unable to climb 4 standard stairs. 2) Climbs 4 standard stairs "marking time" (climbs 1 foot at a time, with both feet on a step before moving to next step), using both arms on 1 or both handrails. 3) Climbs 4 standard stairs "marking time" using 1 arm on 1 handrail. 4) Climbs 4 standard stairs "marking time" not needing handrail. 5) Climbs 4 standard stairs alternating feet, needs handrail for support. 6) Climbs 4 standard stairs alternating feet,
Baseline
Group
Value
95% CI
Ataluren
3.58
± 1.717
Change from Baseline at Week 48
Group
Value
95% CI
Ataluren
-0.46
± 1.141
Change from Baseline at Week 60
Group
Value
95% CI
Ataluren
-0.48
± 1.365
Change From Baseline in Descending 4 Stairs as Assessed by Method Scores During Timed Function TestsSecondary· Baseline, Week 48 and Week 60
Timed test evaluations included scoring of method that ambulatory participants used to complete test. Scale for method used to descend 4 stairs: 1) Unable to descend 4 standard stairs. 2) Descends 4 standard stairs "marking time" (climbs 1 foot at a time, with both feet on a step before moving to next step), using both arms on 1 or both handrails. 3) Descends 4 standard stairs "marking time", using 1 arm on 1 handrail. 4) Descends 4 standard stairs "marking time", not needing handrail. 5) Descends 4 standard stairs alternating feet in both directions, needs handrail for support. 6) Descends 4
Baseline
Group
Value
95% CI
Ataluren
3.63
± 1.740
Change from Baseline at Week 48
Group
Value
95% CI
Ataluren
-0.04
± 1.160
Change from Baseline at Week 60
Group
Value
95% CI
Ataluren
-0.10
± 0.889
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by MyometrySecondary· Baseline, Week (Wk) 48 and Wk 60
Upper extremity myometry was performed using a hand-held dynamometer following standardized procedures. With this system, evaluators judged the strength of each muscle using an 11-point descriptive scoring system. From the individual muscle-group scores, a total composite score was derived. Bilateral assessments were done, and 3 measurements were recorded from each muscle group on each side, when possible. The best of the 3 replicates was used in the analysis. An increase from Baseline is reflective of increased muscle strength, whereas a decrease from Baseline is reflective of decreased muscl
Left (L) Knee Flexion, Baseline
Group
Value
95% CI
Ataluren
9.48
± 4.395
L Knee Flexion, Change from Baseline at Wk 48
Group
Value
95% CI
Ataluren
1.23
± 2.715
L Knee Flexion, Change from Baseline at Wk 60
Group
Value
95% CI
Ataluren
0.64
± 3.079
Right (R) Knee Flexion, Baseline
Group
Value
95% CI
Ataluren
10.15
± 5.439
R Knee Flexion, Change from Baseline at Wk 48
Group
Value
95% CI
Ataluren
0.81
± 2.254
R Knee Flexion, Change from Baseline at Wk 60
Group
Value
95% CI
Ataluren
-0.05
± 2.240
L Knee Extension, Baseline
Group
Value
95% CI
Ataluren
8.63
± 5.124
L Knee Extension, Change from Baseline at Wk 48
Group
Value
95% CI
Ataluren
-1.07
± 1.842
Change in Resting, Active, and Recovery Heart Rate as Assessed by Heart Rate Monitoring With the Polar RS400Secondary· Baseline, Week 48 and Week 60
Heart rate was measured with a Polar RS400 heart rate monitor, which consists of a transmitter strap worn around the chest and a wristwatch receiver. The monitor produces a digital text file with 1 value per minute that represents the mean heart rate for that minute. Mean heart rate values were collected before, during, and after the 6MWT. The participant rested for 5 minutes in a sitting position before the 6MWT, and the mean heart rate for the last minute of this rest period was obtained and documented as the resting heart rate. During the 6MWT, the mean heart rate was collected and document
Resting Heart Rate, Baseline
Group
Value
95% CI
Ataluren
106.46
± 11.163
Resting Heart Rate, Change from Baseline at Wk 48
Group
Value
95% CI
Ataluren
8.30
± 12.382
Resting Heart Rate, Change from Baseline at Wk 60
Group
Value
95% CI
Ataluren
5.16
± 11.197
Active Heart Rate, Baseline
Group
Value
95% CI
Ataluren
152.17
± 13.457
Active Heart Rate, Change from Baseline at Wk 48
Group
Value
95% CI
Ataluren
3.86
± 12.365
Active Heart Rate, Change from Baseline at Wk 60
Group
Value
95% CI
Ataluren
-2.84
± 17.238
Recovery Heart Rate, Baseline
Group
Value
95% CI
Ataluren
113.13
± 12.664
Recovery Heart Rate, Change from Baseline at Wk 48
Group
Value
95% CI
Ataluren
4.64
± 9.079
Change From Baseline in Verbal Memory and Attention as Assessed by the Digit Span TaskSecondary· Baseline, Week 48 and Week 60
The digit span task is a 2-part (forward and backward) test in which a series of digits (3 to 9) were presented to participant in an auditory format only. For forward condition, the participant was to repeat digits back in the order they were presented. For backward condition, the participant was to reverse the order of presentation. Maximum score for each part (digit forward and digit backward) of task is 14; participants received a score of 2 points if they passed both trials, score of 1 point if they passed only 1 trial, and score of 0 points if they failed both trials. A raw score of total
Recalled Forward, Baseline
Group
Value
95% CI
Ataluren
3.66
± 2.378
Recalled Forward, Change from Baseline at Wk 48
Group
Value
95% CI
Ataluren
0.14
± 1.353
Recalled Forward, Change from Baseline at Wk 60
Group
Value
95% CI
Ataluren
0.04
± 1.453
Recalled Backward, Baseline
Group
Value
95% CI
Ataluren
3.15
± 2.524
Recalled Backward, Change from Baseline at Wk 48
Group
Value
95% CI
Ataluren
0.74
± 1.442
Recalled Backward, Change from Baseline at Wk 60
Group
Value
95% CI
Ataluren
0.74
± 1.442
Change From Baseline in Participant-Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) InventorySecondary· Baseline, Week 48 and Week 60
The PedsQL Generic Core Scales comprises 23 questions, which are grouped into 4 scales (physical functioning, emotional functioning, social functioning, and school functioning); the fatigue-specific module included an additional 18 questions. The appropriate age-specific version was completed. On PedsQL Generic Core Scales, items were reversed scored and linearly transformed to a 0 to 100 scale so that higher scores indicate a better HRQL (0=100, 1=75, 2=50, 3=25, and 4=0). Mean is the sum of the items over the number of items that were answered (which accounts for missing data) to create scal
Physical Functioning, Baseline
Group
Value
95% CI
Ataluren
45.13
± 21.114
Physical Functioning Change from Baseline at Wk 48
Group
Value
95% CI
Ataluren
3.16
± 20.180
Physical Functioning Change from Baseline at Wk 60
Group
Value
95% CI
Ataluren
6.25
± 23.545
Emotional Functioning, Baseline
Group
Value
95% CI
Ataluren
65.29
± 16.141
Emotional Function, Change from Baseline at Wk 48
Group
Value
95% CI
Ataluren
8.24
± 18.294
Emotional Function, Change from Baseline at Wk 60
Group
Value
95% CI
Ataluren
12.22
± 17.614
Social Functioning, Baseline
Group
Value
95% CI
Ataluren
61.80
± 16.914
Social Functioning, Change from Baseline at Wk 48
Group
Value
95% CI
Ataluren
4.82
± 19.463
Change From Baseline in Parent- or Caregiver-Reported HRQL as Measured by the PedsQL InventorySecondary· Baseline, Week 48 and Week 60
The PedsQL Generic Core Scales comprises 23 questions, which are grouped into 4 scales (physical functioning, emotional functioning, social functioning, and school functioning); the fatigue-specific module included an additional 18 questions. The appropriate age-specific version was completed. On PedsQL Generic Core Scales, items were reversed scored and linearly transformed to a 0 to 100 scale so that higher scores indicate a better HRQL (0=100, 1=75, 2=50, 3=25, and 4=0). Mean is the sum of the items over the number of items that were answered (which accounts for missing data) to create scal
Physical Functioning, Baseline
Group
Value
95% CI
Ataluren
48.07
± 21.111
Physical Functioning Change from Baseline at Wk 48
Group
Value
95% CI
Ataluren
2.31
± 20.284
Physical Functioning Change from Baseline at Wk 60
Group
Value
95% CI
Ataluren
1.85
± 19.366
Emotional Functioning, Baseline
Group
Value
95% CI
Ataluren
69.45
± 17.465
Emotional Function, Change from Baseline at Wk 48
Group
Value
95% CI
Ataluren
5.27
± 19.595
Emotional Function, Change from Baseline at Wk 60
Group
Value
95% CI
Ataluren
3.29
± 20.021
Social Functioning, Baseline
Group
Value
95% CI
Ataluren
57.95
± 19.397
Social Functioning, Change from Baseline at Wk 48
Group
Value
95% CI
Ataluren
9.38
± 16.956
Adverse events — posted to ClinicalTrials.gov
Time frame: Baseline up to Week 89.
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Duchenne muscular dystrophy (DMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation, is the cause of DMD in approximately 10-15% of boys with the disease. Ataluren is an orally-delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 2a extension trial that will evaluate the long-term safety of ataluren in boys with nonsense mutation DMD, as determined by adverse events and laboratory abnormalities. The study will also assess changes in walking, muscle function, strength, and other important clinical and laboratory measures.
Publications & conference data
5 peer-reviewed publications reference this trial (live from Europe PMC):
NCT04336826 — A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With N
· Phase 2
· completed
NCT03796637 — A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have
· Phase 2
· completed
NCT04117880 — A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia
· Phase 2
· withdrawn
NCT03648827 — A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
· Phase 2
· completed
NCT03179631 — Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
· Phase 3
· completed
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by PTC Therapeutics
Last refreshed: 29 October 2020
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT00759876.